Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease
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|ClinicalTrials.gov Identifier: NCT00012545|
Recruitment Status : Recruiting
First Posted : March 12, 2001
Last Update Posted : March 23, 2021
|First Submitted Date||March 10, 2001|
|First Posted Date||March 12, 2001|
|Last Update Posted Date||March 23, 2021|
|Actual Study Start Date||November 1, 2001|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures
||To procure umbilical cord blood (UCB) from newborns at risk for sickle cell disease, sickle cell trait, and related disorders as well as normal newborns, as controls [ Time Frame: End of study ]
To evaluate the feasibility of performing directed donor umbilical cord blood banking for families at risk for having children with congenital diseases amenable to treatment by autologous gene therapy or allogeneic hematopoietic transplantation; in this protocol, the feasibility will be studied specifically in families with risk for sickle cell anemia and related syndromes.
|Original Primary Outcome Measures||Not Provided|
|Current Secondary Outcome Measures||Not Provided|
|Original Secondary Outcome Measures||Not Provided|
|Current Other Pre-specified Outcome Measures||Not Provided|
|Original Other Pre-specified Outcome Measures||Not Provided|
|Brief Title||Collection and Storage of Umbilical Cord Stem Cells for Treatment of Sickle Cell Disease|
|Official Title||Collection and Storage of Umbilical Cord Hematopoietic Stem Cells for Sickle Cell Disease Therapy|
This study will determine the best ways to collect, process and store umbilical cord blood from babies with sickle cell disease, sickle cell trait and unaffected babies. Sickle cell disease is an abnormality of the hemoglobin in red blood cells that causes the cells to change shape and clump together, preventing their normal flow in the bloodstream. This impairs blood flow to various organs, and the resulting oxygen deprivation causes organ damage.
Cord blood is rich in stem cells (cells produced in the bone marrow that mature to different types of blood cells), which may prove useful in new sickle cell therapies. However, cord blood from babies with sickle cell trait, sickle cell disease and normal babies may act differently under laboratory conditions, so it is important to learn how best to work with blood from all three groups of babies for future use in possible treatments.
Pregnant women between 18 and 45 years of age who are at risk of having an infant with sickle cell disease and normal volunteers who are pregnant and not at risk for this disease may be eligible for this study. Potential participants will be counseled about donating her infant s blood in order to make an informed choice.
All women who participate in the study will provide a medical history and have blood collected from the umbilical cord and placenta (afterbirth) after the baby s delivery. The blood will be tested for various infectious diseases, processed, frozen and stored for research purposes. In addition, blood from women with babies at risk for sickle cell disease will be tested for the presence of the sickle cell gene, tissue typed, and used for research as follows:
Participants and their family doctor or the baby s pediatrician will be contacted twice a year for information about changes in the baby s health. Participants may also be asked permission to perform new tests developed by researchers.
|Detailed Description||Umbilical cord blood is a source of hematopoietic stem cells (HSCs) for transplantation or gene therapy. Our goal is to procure umbilical cord blood (UCB) from newborns at risk for sickle cell disease, sickle cell trait, and related disorders as well as normal newborns and store clinical grade cord blood units (CBU) for future use in clinical transplantation or gene therapy. Cord blood units will be collected from an indefinite number of subjects until 50 CBU from newborns with homozygous sickle cell disease have been cryopreserved. Maternal subjects will be identified as being at risk to have an infant with sickle cell disease, will be between the ages of 18 and 45 years, and will meet specified medical history criteria. Mothers will have to deliver at INOVA Fairfax Hospital (collection provider site) and the CB will be collected by NCBP collectors ex utero, will be transported to the NCBP CB facility and will be processed, tested and stored. Frozen CBU will be transferred to the NIH for clinical transplantation or gene therapy studies upon NIH request.|
|Study Design||Observational Model: Case-Only
Time Perspective: Cross-Sectional
|Target Follow-Up Duration||Not Provided|
|Sampling Method||Non-Probability Sample|
|Study Population||This is a pilot feasibility study for which umbilical cord blood samples will be collected and transported to the NIH Clinical Center for our developmental research. Pregnant women whose babies are at risk for sickle cell anemia will be identified and referred to the NIH Research Coordinator for evaluation and entry into the study. The NIH Research Coordinator is experienced in performing patient education and counseling concerning sickle cell disease, and obtaining informed consent.|
|Study Groups/Cohorts||Healthy Pregnant Volunteers
Pregnant women whose babies are at risk for sickle cell anemia will be identified and referred to the NIH Research Coordinator for evaluation and entry into the study.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Study Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
Pregnant women who are at risk of having an infant with sickle cell anemia related diseases.
The types of sickle cell disease include the following:
|Ages||up to 45 Years (Child, Adult)|
|Accepts Healthy Volunteers||Yes|
|Listed Location Countries||United States|
|Removed Location Countries|
|Other Study ID Numbers||010122
|Has Data Monitoring Committee||Not Provided|
|U.S. FDA-regulated Product||Not Provided|
|IPD Sharing Statement||Not Provided|
|Responsible Party||National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )|
|Study Sponsor||National Heart, Lung, and Blood Institute (NHLBI)|
|PRS Account||National Institutes of Health Clinical Center (CC)|
|Verification Date||October 9, 2020|